Posts by: Alice Melão

News

Abeona Therapeutics is planning to expand its ongoing ABO-102 gene therapy Phase 1/2 trial to include patients with Sanfilippo syndrome type A (MPS IIIA) at earlier stages of the ... Read more

December 13, 2018December 13, 2018

New Stem Cell Lines Derived From MPSIIIB Patient May Help Improve Preclinical Research

News

Two new experimental stem cell lines derived from a patient with mucopolysaccharydosis IIIB (MPSIIIB), also known as Sanfilippo syndrome type B, were developed by Spanish researchers. These stem cells ... Read more

November 8, 2018November 8, 2018

Shire’s Investigational ERT Shows Limited Benefits for Sanfilippo Type A Children in Phase 2b Trial

News

Administration of an engineered version of the heparan-N-sulfatase (HNS) enzyme into the cerebrospinal fluid directly through the spinal canal failed to improve neurocognitive activity in infants with early-stage Sanfilippo syndrome ... Read more

September 25, 2018September 25, 2018

More Studies Needed to Characterize Features, Course of MPS III, Review Says

News

Very little is still known about the clinical features and natural course of Sanfilippo syndrome, also known as mucopolysaccharidosis (MPS) III, a rare disease that can significantly affect children’s ... Read more

September 13, 2018September 13, 2018

Metabolomics Study Helps Researchers Get Closer to Identifying Sanfilippo Biomarkers

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A comprehensive analysis of compounds present in the urine of patients with Sanfilippo syndrome provides new insight into the biological processes likely involved in the disease. The research could help ... Read more

June 27, 2018June 27, 2018

Common Sugar, a Food Additive, Seen to Ease Brain Inflammation in Mouse Model of Sanfilippo IIIB

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A common sugar often used as a food additive was seen to ease inflammation in the brain and retina, and extend the life of Sanfilippo disease IIIB (mucopolysaccharidosis IIIB) mice in ... Read more

March 15, 2018March 15, 2018

Researchers Review Recent Advances in Sanfilippo Syndrome Therapies

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Many efforts have been made to improve care and find a cure for patients with Sanfilippo disease. While some pursuits have fallen short, others hold promise. In the study ... Read more

February 6, 2018February 6, 2018

Sobi’s Enzyme Replacement Therapy for Sanfilippo Syndrome Type A to Enter Clinical Trials

News

The U.S. Food and Drug Administration (FDA) has accepted an investigational new drug (IND) application for Swedish Orphan Biovitrum AB‘s (Sobi) enzyme replacement therapy SOBI003, for the treatment of Sanfilippo syndrome ... Read more

January 3, 2018January 3, 2019

Heparan Sulfate Buildup Not Directly Linked to MPSIIIA Symptom Onset, Mouse Study Shows

News

Heparan sulfate accumulation is not a good indicator of Sanfilippo syndrome type A symptom onset, a new mouse model of the disease suggests. This finding, reported in the study, ... Read more

Posts by: Alice Melão

Alice Melão

Abeona Will Include Early-stage MPS IIIA Patients in ABO-102 Gene Therapy Trial

New Stem Cell Lines Derived From MPSIIIB Patient May Help Improve Preclinical Research

Shire’s Investigational ERT Shows Limited Benefits for Sanfilippo Type A Children in Phase 2b Trial

More Studies Needed to Characterize Features, Course of MPS III, Review Says

Metabolomics Study Helps Researchers Get Closer to Identifying Sanfilippo Biomarkers

Common Sugar, a Food Additive, Seen to Ease Brain Inflammation in Mouse Model of Sanfilippo IIIB

Researchers Review Recent Advances in Sanfilippo Syndrome Therapies

Sobi’s Enzyme Replacement Therapy for Sanfilippo Syndrome Type A to Enter Clinical Trials

Heparan Sulfate Buildup Not Directly Linked to MPSIIIA Symptom Onset, Mouse Study Shows

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